Acceleration of retarded growth in children with disease after treatment with alglucerase

P. Kaplan*, A. Mazur, O. Manor, J. Charrow, J. Esplin, T. J. Gribble, R. S. Wappner, J. S. Wisch, N. J. Weinreb

*Corresponding author for this work

Research output: Contribution to journalArticlepeer-review

90 Scopus citations


Objectives: The incidence and severity of growth retardation in children with type 1 Gaucher disease and the response to enzyme replacement therapy with alglucerase were studied. Study design: A retrospective analysis of growth in 99 children and adolescents with type 1 Gaucher disease before treatment, and in 54 of those subjects during treatment, was done. Growth was compared with gender, age, and dosage of replacement enzyme. Results: Linear growth was normal in the first 1 to 2 years of life and then decelerated. Height was at or below the 5th percentile in 50% of all subjects immediately before treatment. The mean z score was -1.49 (95% confidence interval, -1.83 to -1.16), corresponding to the 6.8th percentile for height. Seventy-two percent were below the 50th percentile and 50% were at or below the 5th percentile for mid-parental height (p <0.001). One and one-half years after treatment was staffed, the estimated mean z score for all subjects was - 1.01, which corresponds to the 16th percentile for height. Normal growth was achieved within 4 to 30 months in eight of nine subjects who were at or below the 5th percentile. It occurred only in those receiving higher doses (60 to 120 U/kg per 4-week period) of alglucerase. There was a significant association between z scores for height before treatment and liver enlargement (r = 0.57; p <0.01). Conclusions: Half of the subjects who manifest type 1 Gaucher disease in childhood have growth retardation. Treatment with adequate amounts of modified enzyme replacement was effective in normalizing linear growth.

Original languageEnglish (US)
Pages (from-to)149-153
Number of pages5
JournalJournal of Pediatrics
Issue number1
StatePublished - 1996

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health


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